Wednesday February 22d, 2017: “Research for rare disease: RD-Connect and the importance of data sharing” Pr. Hanns Lochmüller, MD, FAAN, Chair of Experimental Myology, Deputy Director, Institute of Genetic Medicine, The John Walton Muscular Dystrophy Research Centre – MRC Centre for Neuromuscular Diseases. Newcastle University , Newcastle upon Tyne- United Kingdom.
Monday January 23rd, 2017: “Rescue of exon skipping defects using modified U1 snRNAs: a novel therapeutic approach for spinal muscular atrophy” Prof. Franco Pagani, Head of Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology(ICGEB), Trieste, Italy.
Wednesday 18th January, 2017: “From viruses to designer nanoparticles – Tailoring adeno-associated viruses for gene therapy” Prof. Dr. Hildegard Büning, Head of the Laboratory for Infection Biology & Gene Transfer in the Institute of Experimental Hematology at Hannover Medical School, Hannover, Germany.
Friday 18th November, 2016: “Gene Editing to Correct Chronic Granulomatous Disease” Prof. Harry Malech, Chief, Laboratory of Host Defenses; Head, Genetic Immunotherapy Section; National Institute of Allergy and Infectious Diseases; National Institutes of Health. Bethesda, MD, USA.
Tuesday 4th October, 2016: “Targeting FVIII expression in endothelial and myeloid cells for hemophilia a gene therapy in mice” Prof. Antonia Follenzi, Associate professor of Histology, Università degli Studi del Piemonte Orientale “Amedeo Avogadro”, Dipartimento di Scienze della Salute (Dept. of Health Sciences) in Novara, Italy.
Tuesday 21th June,2016: “Du génome au métabolite” Marcel Salanoubat, Directeur de l’UMR 8030, « Génomique Métabolique », Genoscope, Evry, France.
Tuesday 14th June 2016: “Gene therapy for liver genetic diseases” Dr Gloria González Aseguinolaza, Director of the Gene Therapy and Regulation of Gene Expression program, Head of the laboratory of Gene therapy for liver diseases, CIMA, University of Navarra, Pamplona, Spain.
Tuesday 7th June 2016: “MIP nanoparticles (“plastic antibodies”) for separation and diagnostic applications” Pr Sergey A. Piletsky, Head of Leicester Biotechnology Group, Leicester Biotechnology Group, Department of Chemistry, University of Leicester, Leicester United Kingdom)
Tuesday 31th May 2016: “In vivo clonal tracking of human hematopoiesis through integrations sites analysis” Dr Luca Biasco, Group Leader at SR-TIGET Milan, Italy
Caroline Le Guiner (Biotherapies Institute of Rare Diseases and Atlantic Gene Therapies) participates in the Myology 2016 to talk about the development of gene therapy products for Duchenne muscular dystrophy. Play video